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WHIM ÁõÈıº ½ÃÀå - ½ÃÀå ÀλçÀÌÆ®, ¿ªÇÐ, ½ÃÀå ¿¹Ãø(2034³â)WHIM Syndrome - Market Insight, Epidemiology, and Market Forecast - 2034 |
ÀÌ º¸°í¼´Â ¹Ì±¹, EU 4°³±¹ ¹× ¿µ±¹(µ¶ÀÏ, ÇÁ¶û½º, ÀÌÅ»¸®¾Æ, ½ºÆäÀÎ), ÀϺ»ÀÇ WHIM, ¿ª»çÀû ¹× ¿¹Ãø ¿ªÇÐ, WHIM ½ÃÀå µ¿Çâ¿¡ ´ëÇÑ »ó¼¼ÇÑ ÀÌÇØ¸¦ Á¦°øÇÕ´Ï´Ù.
¶ÇÇÑ, ÇöÀç Ä¡·á¹ý, ½Å¾à, °³º° Ä¡·á¹ýÀÇ ½ÃÀå Á¡À¯À², 2020³â¿¡¼ 2034³â±îÁö ÁÖ¿ä 7°³±¹ WHIM ½ÃÀå ±Ô¸ð ÇöȲ ¹× ¿¹ÃøÀ» Á¦°øÇÕ´Ï´Ù. ¶ÇÇÑ, ÇöÀç WHIM Ä¡·á °üÇà, ½ÃÀå ÃËÁø¿äÀÎ, ½ÃÀå À庮, SWOT ºÐ¼®, »óȯ ¹× ½ÃÀå Á¢±Ù¼º, ¹ÌÃæÁ· ÀÇ·á ¼ö¿ä µîÀ» ´Ù·ç¸ç, ÃÖ»óÀÇ ±âȸ¸¦ ¼±º°ÇÏ°í ½ÃÀå ÀáÀç·ÂÀ» Æò°¡ÇÕ´Ï´Ù.
Á¶»ç ±â°£ : 2020-2034³â
WHIM ÁõÈıº Ä¡·á ½ÃÀå
WHIM ÁõÈıºÀº CXCR4 À¯ÀüÀÚÀÇ µ¹¿¬º¯ÀÌ·Î ÀÎÇØ ¹ß»ýÇÏ´Â Èñ±ÍÇÑ À¯Àüº´À¸·Î ¸é¿ª °áÇÌ, »ç¸¶±Í, Àú°¨¸¶±Û·ÎºÒ¸°Ç÷ÁõÀÇ ¼¼ °¡Áö Áõ»óÀ» À¯¹ßÇϸç, Á¾Á¾ °ñ¼öÀÌ¿µ¾çÁõ(È£Áß±¸°¡ °ñ¼ö¿¡ Æ÷ȹµÇ´Â »óÅÂ)À» µ¿¹ÝÇÕ´Ï´Ù. WHIMÀº ÀÌ ÁõÈıºÀÇ ÁÖ¿ä Áõ»óÀÎ »ç¸¶±Í, Àú°¨¸¶±Û·ÎºÒ¸°Ç÷Áõ, °¨¿°, °ñ¼öÀÌÇü¼ºÁõÀÇ ¸Ó¸®±ÛÀÚ¸¦ µý °ÍÀ¸·Î, ÀÌ ÁõÈıºÀº ¸é¿ª±â´ÉÀ» ÇöÀúÇÏ°Ô ÀúÇϽÃÄÑ °¨¿°¿¡ ½±°Ô °É¸®°Ô ÇÕ´Ï´Ù.
WHIM ÁõÈıºÀº ¸î °¡Áö Ư¡ÀûÀÎ Áõ»óÀ» º¸À̴µ¥, WHIM ÁõÈıº ȯÀÚ´Â ¸é¿ª·ÂÀÌ ¾àÇØÁ® ¸é¿ª±â´ÉÀÌ ÀúÇÏµÇ¾î °¨¿°¿¡ Ãë¾àÇØÁý´Ï´Ù. ¶Ç ´Ù¸¥ µÎµå·¯Áø Áõ»óÀº ÇÇºÎ¿Í Á¡¸·¿¡ Áö¼ÓÀûÀÌ°í ¹Ýº¹ÀûÀÎ »ç¸¶±Í°¡ »ý±â´Â °ÍÀÔ´Ï´Ù. Àú°¨¸¶±Û·ÎºÒ¸°Ç÷ÁõÀº Ç÷¾× ³» ¸é¿ª±Û·ÎºÒ¸°(Ç×ü) ¼öÄ¡°¡ °¨¼ÒÇÏ¿© ¸é¿ª°è ±â´É Àå¾Ö¸¦ ´õ¿í ¾ÇȽÃŰ´Â °ÍÀÌ Æ¯Â¡ÀÔ´Ï´Ù. ¶ÇÇÑ, °ñ¼ö¿¡ È£Áß±¸°¡ ÃàÀûµÇÁö¸¸ Ç÷·ù¿¡¼´Â °¨¼ÒÇÏ´Â °ñ¼öÀÌÇü¼ºÁõÀº WHIM ÁõÈıºÀÇ Áß¿äÇÑ Æ¯Â¡ÀÔ´Ï´Ù. ÀÌ·¯ÇÑ Áõ»óÀº CXCR4 À¯ÀüÀÚÀÇ µ¹¿¬º¯ÀÌ·Î ÀÎÇØ ¹ß»ýÇϸç, °¨¿°¿¡ È¿°úÀûÀ¸·Î ´ëÀÀÇϰí Àü¹ÝÀûÀÎ °Ç°À» À¯ÁöÇÏ´Â ½ÅüÀÇ ´É·Â¿¡ ¿µÇâÀ» ¹ÌĨ´Ï´Ù.
WHIM ÁõÈıºÀÇ Áø´ÜÀº Àç¹ß¼º °¨¿°, »ç¸¶±Í, Àú°¨¸¶±Û·ÎºÒ¸°Ç÷Áõ, °ñ¼öÀÌÇü¼ºÁõ µîÀÇ Áõ»ó¿¡ ´ëÇÑ ÀÓ»óÀû Æò°¡¿Í CXCR4 À¯ÀüÀÚÀÇ µ¹¿¬º¯À̸¦ È®ÀÎÇϱâ À§ÇÑ À¯ÀüÀÚ °Ë»ç°¡ ÇÊ¿äÇÕ´Ï´Ù. À¯ÀüÀÚ °Ë»ç´Â È®Áø Áø´ÜÀ» À§ÇØ ¸Å¿ì Áß¿äÇÏÁö¸¸, ÀÌ °úÁ¤¿¡´Â ¹ÌÃæÁ· ¼ö¿ä°¡ ÀÖ½À´Ï´Ù. Áõ»óÀÌ ´Ù¸¥ Áúȯ°ú Áߺ¹µÉ ¼ö Àֱ⠶§¹®¿¡ ¸¹Àº ȯÀÚµéÀÌ ÀÎ½Ä ºÎÁ·°ú °ú¼Ò Áø´ÜÀ¸·Î ÀÎÇØ Áø´ÜÀÌ ´Ê¾îÁö´Â °æ¿ì°¡ ¸¹½À´Ï´Ù.
WHIM ÁõÈıºÀÇ Á¶±â °³ÀÔ°ú È¿°úÀûÀÎ °ü¸®¸¦ À§Çؼ´Â ÀÎ½Ä Á¦°í, À¯ÀüÀÚ °Ë»ç¿¡ ´ëÇÑ Æø³ÐÀº Á¢±Ù¼º, Áø´Ü µµ±¸ÀÇ °È¸¦ ÅëÇØ ÀÌ·¯ÇÑ °ÝÂ÷¸¦ ÇØ¼ÒÇÏ´Â °ÍÀÌ ÇʼöÀûÀÔ´Ï´Ù.
WHIM ÁõÈıºÀÇ Ä¡·á´Â Áõ»ó °ü¸®¿Í ±Ùº»ÀûÀÎ À¯ÀüÀÚ ÀÌ»ó¿¡ ´ëÇÑ ´ëó¿¡ ÃÊÁ¡À» ¸ÂÃß°í ÀÖ½À´Ï´Ù. ÇöÀç Ä¡·á¹ýÀ¸·Î´Â È£Èí±âÁúȯ¿¡ ´ëÇÑ Áö¼ÓÀû ¾ç¾ÐÈ£Èí¿ä¹ý(CPAP), °¨¿° ¹× ¸é¿ªºÎÀü¿¡ ´ëÇÑ ¾à¹° Ä¡·á µîÀÌ ÀÖÀ¸¸ç, XOLREMDI(mavorixafor)¿Í °°Àº CXCR4 ±æÇ×Á¦´Â CXCR4 ¼ö¿ëü¸¦ ¾ïÁ¦ÇÏ¿© ±Ùº»ÀûÀÎ ¿øÀÎÀ» °Ü³ÉÇÏ¿© ¸é¿ª±â´ÉÀ» °³¼±Çϰí Áõ»óÀ» ¿ÏÈÇÒ ¼ö ÀÖ½À´Ï´Ù. ¸é¿ª±â´ÉÀ» °³¼±Çϰí Áõ»óÀ» ¿ÏȽÃų ¼ö ÀÖ½À´Ï´Ù. ¶ÇÇÑ, À¯ÀüÀÚ º¯À̸¦ ±³Á¤ÇÏ´Â Àå±âÀûÀÎ ÇØ°áÃ¥À¸·Î À¯ÀüÀÚ Ä¡·á°¡ °ËÅäµÇ°í ÀÖ½À´Ï´Ù.
±×·¯³ª CPAP ¹× °¨¿° °ü¸®¿Í °°Àº ÇöÀç Ä¡·á¹ýÀ» ÁؼöÇÏ´Â °ÍÀº ȯÀڵ鿡°Ô ¾î·Á¿î ÀÏÀ̸ç, ÇöÀç »ç¿ë °¡´ÉÇÑ ¸¹Àº Ä¡·á¹ýÀº ÀÌ ÁõÈıºÀÇ º¹ÀâÇÑ Áõ»ó¿¡ ºÎºÐÀûÀ¸·Î¸¸ ´ëÀÀÇϰí ÀÖÀ¸¸ç, °³º°ÈµÈ Ä¡·á ¿É¼ÇÀÌ ºÎÁ·ÇÕ´Ï´Ù. WHIM ÁõÈıº ȯÀÚÀÇ ¿¹ÈÄ¿Í »îÀÇ ÁúÀ» °³¼±Çϱâ À§Çؼ´Â È¿°úÀûÀÎ ¸ÂÃã Ä¡·á¿¡ ´ëÇÑ ´õ ¸¹Àº ¿¬±¸¿Í »õ·Î¿î Ä¡·á¹ý¿¡ ´ëÇÑ Á¢±Ù¼º °³¼±ÀÌ ÇʼöÀûÀÔ´Ï´Ù.
º» º¸°í¼ÀÇ WHIM ¿ªÇÐ Àå¿¡¼´Â 2020³âºÎÅÍ 2034³â±îÁö ¹Ì±¹, EU 4°³±¹(µ¶ÀÏ, ÇÁ¶û½º, ÀÌÅ»¸®¾Æ, ½ºÆäÀÎ), ¿µ±¹, ÀϺ» µî ÁÖ¿ä 7°³±¹À» ´ë»óÀ¸·Î WHIM ÁõÈıº Áø´Ü À¯º´ÀÚ ¼ö, ¼ºº°¿¡ µû¸¥ WHIM ÁõÈıº Áø´Ü À¯º´ÀÚ ¼ö, ¿¬·Éº° WHIM ÁõÈıº Áø´Ü À¯º´ÀÚ ¼ö, ¿¬·É¿¡ µû¸¥ WHIM ÁõÈıºÀÇ Áø´Ü À¯º´ÀÚ ¼ö¸¦ ¼¼ºÐÈÇÏ¿© °ú°Å ¿ªÇÐ ¹× ¿¹ÃøÀ» º¸¿©ÁÝ´Ï´Ù.
XOLREMDI(mavorixafor) : X4 Pharmaceuticals
XOLREMDI(mavorixafor)´Â ¼±ÅÃÀû CXCR4 ¼ö¿ëü ±æÇ×Á¦·Î ÀÛ¿ëÇÏ´Â ÃÖÃÊÀÇ °æ±¸¿ë ÀúºÐÀÚ ¾à¹°·Î, WHIM ÁõÈıº ȯÀÚ¸¦ À§ÇÑ ÃÖÃÊÀÇ FDA ½ÂÀΠĸ½¶ÀÔ´Ï´Ù. ¸¶º¼¸¯»çÆúÀº °ñ¼ö¿¡¼ ¹éÇ÷±¸ÀÇ µ¿¿ø°ú ¼ö¼ÛÀ» ÃËÁøÇϸç, WHIM ÁõÈıº ¿Ü¿¡µµ ÁßÁõ ¼±Ãµ¼º È£Áß±¸°¨¼ÒÁõ(SCN), ¿Í¸£µ§½´Æ®·½ °Å´ë±Û·ÎºÒ¸°Ç÷Áõ, À¯¹æ¾Ï, ¾Ç¼º Èæ»öÁ¾, ¼¼´¢°ü ½Å¼¼Æ÷¾Ï(ccRCC)¿¡ ´ëÇÑ ÀûÀÀÁõÀ¸·Î °ËÅäµÇ°í ÀÖ½À´Ï´Ù.
XOLREMDI´Â ±ÔÁ¦ ´ç±¹ÀÇ ¸¶ÀϽºÅæÀ¸·Î Å« ÁÖ¸ñÀ» ¹Þ°í ÀÖ½À´Ï´Ù. ¹Ì±¹ FDA´Â ¼ºÀÎ WHIM ÁõÈıº Ä¡·áÁ¦·Î 2020³â 10¿ù Fast Track Designation(FTD), 2019³â 11¿ù Breakthrough Therapy Designation(BTD)À» ½ÂÀÎÇß½À´Ï´Ù. ¶ÇÇÑ, 2018³â 10¿ù¿¡´Â FDA·ÎºÎÅÍ Èñ±ÍÁúȯ Ä¡·áÁ¦ ÁöÁ¤(ODD)À», 2020³â 12¿ù¿¡´Â Èñ±Í ¼Ò¾ÆÁúȯ ÁöÁ¤(ODD)À» ¹Þ¾Ò½À´Ï´Ù. À¯·´¿¬ÇÕ ÁýÇàÀ§¿øÈ¸µµ 2019³â 7¿ù WHIM ÁõÈıº¿¡ ´ëÇØ ODD¸¦ ºÎ¿©Çß½À´Ï´Ù.
XOLREMDI´Â 2024³â 60%ÀÇ Á¡À¯À²À» ´Þ¼ºÇÒ °ÍÀ¸·Î ¿¹»óµÇ¸ç, Á¤Á¡¿¡ µµ´ÞÇϱâ±îÁö 6³âÀÌ ¼Ò¿äµÉ °ÍÀ¸·Î ¿¹»óµÇ¸ç, Áß¼ÓÀ¸·Î Èí¼öµÉ °ÍÀ¸·Î º¸ÀÔ´Ï´Ù.
¹Ì±¹ FDAÀÇ ½ÂÀÎÀ» ¹ÞÀº ÇÁ·¹¸¯»çÆ÷¸£(MOZOBIL, AMD3100)´Â ºñÈ£ÁöŲ¸²ÇÁÁ¾ ¹× ´Ù¹ß¼º °ñ¼öÁ¾ ȯÀÚÀÇ ÀÚ°¡ À̽ÄÀ» À§ÇØ CD34 Á¶Ç÷¸ð¼¼Æ÷¸¦ µ¿¿øÇÏ´Â Ä¡·áÁ¦ÀÔ´Ï´Ù. ¶ÇÇÑ, CXCR4¸¦ ºÎºÐÀûÀ¸·Î ¾ïÁ¦ÇÏ¿© WHIM ÁõÈıº¿¡ ´ëÀÀÇϸç, Áٱ⼼Æ÷ µ¿¿øº¸´Ù ³·Àº ¿ë·®À¸·Î »ç¿ëµË´Ï´Ù. ¹Ì±¹ ±¹¸³¾Ë·¹¸£±â°¨¿°º´¿¬±¸¼Ò´Â WHIM ÁõÈıº¿¡ ´ëÇÑ plerixaforÀÇ °³¹ßÀ» ÁøÇàÇϰí ÀÖÀ¸¸ç, ÀÓ»ó 3»ó ½ÃÇèÀ» ¿Ï·áÇÏ¿´½À´Ï´Ù. ±× ±âÀüÀº CXCR4 ¼ö¿ëü¸¦ ƯÀÌÀûÀÌ°í °¡¿ªÀûÀ¸·Î ¾ïÁ¦ÇÏ¿© °£Áú¼¼Æ÷À¯·¡ÀÎÀÚ-1(SDF-1)ÀÇ °áÇÕÀ» ¾ïÁ¦ÇÏ°í ¼¼Æ÷ Á¢Âø¿¡ ¿µÇâÀ» ¹ÌÄ£´Ù´Â °ÍÀÔ´Ï´Ù.
±×·¯³ª 2021³â ÀÓ»ó II/III»ó ½ÃÇèÀÌ Á¾·áµÈ ÀÌÈÄ, ÀÌ ¾à¿¡ ´ëÇÑ ÃֽŠÁ¤º¸³ª Ãß°¡ ÁøÀüÀÌ º¸°íµÇÁö ¾Ê°í ÀÖ½À´Ï´Ù. ÀÌ·¯ÇÑ Á¤Ã¼´Â °³¹ß ÆÄÀÌÇÁ¶óÀΰú ±ÔÁ¦ »óȲ¿¡¼ °³¹ßÀÌ Áߴܵǰųª ÁøÀüÀÌ ¾øÀ» ¼ö ÀÖÀ½À» ½Ã»çÇÕ´Ï´Ù.
2024³â WHIM ÁõÈıº¿¡ ´ëÇÑ Ã¹ ¹øÂ° Ä¡·áÁ¦°¡ ½ÂÀÎµÇ¸é¼ WHIMÀÇ ½ÃÀå Àü¸ÁÀº È®´ëµÉ °ÍÀ¸·Î ¿¹»óµË´Ï´Ù.
WHIM ÁõÈıºÀÇ Ä¡·á¿¡´Â ¾à¸®ÇÐÀû Á¢±Ù°ú ºñ¾à¸®ÇÐÀû Á¢±ÙÀÌ ¸ðµÎ Æ÷ÇԵ˴ϴÙ. ¾à¸®ÇÐÀû Ä¡·á¿¡´Â ÁÖ·Î CXCR4 ½ÅÈ£Àü´ÞÀ» ºÎºÐÀûÀ¸·Î Â÷´ÜÇÏ¿© È£Áß±¸°¨¼ÒÁõ °ü¸®¿¡ µµ¿òÀ» ÁÖ´Â CXCR4 ¼ö¿ëü ±æÇ×Á¦ÀÎ ÇÁ·¹¸¯»çÆ÷¸£(MOZOBIL)°¡ Æ÷ÇԵ˴ϴÙ. ÀÌ´Â ¹éÇ÷±¸ µ¿¿øÀ» ÃËÁøÇϱâ À§ÇØ °ú¸³±¸ Áý¶ô ÀÚ±Ø ÀÎÀÚ(G-CSF)¿Í º´¿ëÇÏ´Â °æ¿ì°¡ ¸¹½À´Ï´Ù. ¶ÇÇÑ, WHIM ÁõÈıºÀÇ Æ¯Â¡ÀûÀÎ ¸é¿ª °áÇÌÀ¸·Î ÀÎÇÑ °¨¿°À» °ü¸®Çϱâ À§ÇØ Ç×»ýÁ¦¿Í Ç×Áø±ÕÁ¦¸¦ ó¹æÇϱ⵵ ÇÕ´Ï´Ù.
¾à¹° Ä¡·á ÀÌ¿ÜÀÇ Ãø¸é¿¡¼´Â °¨¿°ÁõÀÇ °ü¸®¿Í ¿¹¹æÀ» À§ÇÑ Á¤±âÀûÀÎ ¸ð´ÏÅ͸µÀ» Æ÷ÇÔÇÑ ÁöÁö¿ä¹ýÀÌ Áß¿äÇÕ´Ï´Ù. À§»ý °ü¸®¸¦ öÀúÈ÷ ÇÏ°í °¨¿° À§ÇèÀ» ÃÖ¼ÒÈÇÏ´Â µîÀÇ »ýȰ½À°ü Á¶Àýµµ ±ÇÀåµË´Ï´Ù. ¶ÇÇÑ, ȯÀÚ°¡ ¸¸¼ºÁúȯÀ» ¾Î°í ÀÖ´Â µ¥ µû¸¥ Á¤¼Àû, ½É¸®Àû ¾î·Á¿ò¿¡ ´ëóÇÒ ¼ö ÀÖµµ·Ï ½É¸®»çȸÀû Áö¿øµµ Áß¿äÇÕ´Ï´Ù. ÀÌ·¯ÇÑ Àü·«ÀÇ Á¶ÇÕÀº WHIM ÁõÈıº ȯÀÚÀÇ Àü¹ÝÀûÀÎ °Ç° °á°ú¿Í »îÀÇ ÁúÀ» °³¼±ÇÏ´Â °ÍÀ» ¸ñÇ¥·Î Çϰí ÀÖ½À´Ï´Ù.
º» º¸°í¼´Â ÁÖ¿ä 7°³±¹ WHIM ÁõÈıº ½ÃÀå¿¡ ´ëÇØ Á¶»çÇßÀ¸¸ç, ½ÃÀå °³¿ä, ¿ªÇÐ, ȯÀÚ µ¿Çâ, »õ·Î¿î Ä¡·á¹ý, 2034³â±îÁöÀÇ ½ÃÀå ±Ô¸ð ¿¹Ãø, ¹ÌÃæÁ· ÀÇ·á ¼ö¿ä µîÀ» Á¶»çÇÏ¿© ÀüÇØµå¸³´Ï´Ù.
DelveInsight's "WHIM Syndrome Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of the WHIM, historical and forecasted epidemiology as well as the WHIM market trends in the United States, EU4 and the UK (Germany, France, Italy, Spain), the United Kingdom, and Japan.
The WHIM market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM WHIM market size from 2020 to 2034. The Report also covers current WHIM treatment practice, market drivers, market barriers, SWOT analysis, reimbursement and market access, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Study Period: 2020-2034
WHIM Syndrome Treatment Market
WHIM Overview
WHIM syndrome is a rare genetic disorder caused by mutations in the CXCR4 gene, leading to a triad of symptoms: immunodeficiency, warts, and hypogammaglobulinemia, often accompanied by myelokathexis, a condition where neutrophils are trapped in the bone marrow. This syndrome significantly impairs immune function and increases susceptibility to infections. The term WHIM is an acronym for the main signs of the syndrome: warts, hypogammaglobulinemia, infections, and myelokathexis.
WHIM syndrome manifests through several distinct symptoms. Individuals with this condition experience immunodeficiency, which leads to increased vulnerability to infections due to compromised immune function. Another prominent symptom is the development of persistent and recurrent warts on the skin and mucous membranes. Hypogammaglobulinemia, characterized by low levels of immunoglobulins (antibodies) in the blood, further exacerbates immune system dysfunction. Additionally, myelokathexis, a condition where neutrophils accumulate in the bone marrow but are reduced in the bloodstream, is a key feature of WHIM syndrome. These symptoms arise from mutations in the CXCR4 gene, impacting the body's ability to effectively respond to infections and maintain overall health.
WHIM Diagnosis
Diagnosing WHIM syndrome involves clinical evaluation of symptoms such as recurrent infections, warts, hypogammaglobulinemia, and myelokathexis, followed by genetic testing to confirm CXCR4 gene mutations. While genetic testing is crucial for a definitive diagnosis, there are unmet needs in this process. Many patients face delays due to lack of awareness and underdiagnosis, as symptoms may overlap with other conditions.
Additionally, the availability of specialized testing and expertise can be limited, further complicating timely diagnosis. Addressing these gaps through improved awareness, broader access to genetic testing, and enhanced diagnostic tools is essential for early intervention and effective management of WHIM syndrome.
WHIM Treatment
Treatment for WHIM syndrome focuses on managing symptoms and addressing the underlying genetic defect. Current approaches include continuous positive airway pressure (CPAP) therapy for associated respiratory issues, and medications to manage infections and immune deficiencies. CXCR4 antagonists, such as XOLREMDI (mavorixafor), aim to target the root cause by inhibiting the CXCR4 receptor, potentially improving immune function and reducing symptoms. Additionally gene therapy is being explored as a long-term solution to correct the genetic mutation.
However, significant unmet needs remain. Adherence to current therapies, such as CPAP and infection management, can be challenging for patients. Additionally, many available treatments only partially address the syndrome's complex symptoms, and there is a lack of personalized therapeutic options. More research into effective, tailored treatments and better access to emerging therapies are critical for improving outcomes and quality of life for individuals with WHIM syndrome.
As the market is derived using the patient-based model, the WHIM epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Diagnosed Prevalent Cases of WHIM Syndrome, Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome, and Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034.
The drug chapter segment of the WHIM report encloses a detailed analysis of WHIM approved and off-label drugs along with late-stage (Phase III) pipeline drugs. It also helps to understand the WHIM clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
WHIM Syndrome Marketed Drugs
XOLREMDI (mavorixafor): X4 Pharmaceuticals
XOLREMDI (mavorixafor) is the first FDA approved capsules indicated in patients with WHIM syndrome promising first-in-class, oral small molecule, functioning as a selective CXCR4 receptor antagonist. This drug enhances the mobilization and trafficking of white blood cells from the bone marrow. Beyond WHIM syndrome, Mavorixafor is being explored for severe congenital neutropenia (SCN), Waldenstrom's macroglobulinemia, breast cancer, malignant melanoma, and clear cell renal cell carcinoma (ccRCC).
In terms of regulatory milestones, XOLREMDI has garnered significant attention. The US FDA granted Fast Track Designation (FTD) in October 2020 and Breakthrough Therapy Designation (BTD) in November 2019 for treating WHIM syndrome in adults. Additionally, it received Orphan Drug Designation (ODD) from the FDA in October 2018 and Rare Pediatric Disease Designation in December 2020. The European Commission also granted ODD for WHIM syndrome in July 2019.
XOLREMDI, according to the DelveInsight's forecast model would capture a peak share of 60% in 2024 and is anticipated to take 6 years to peak with a medium-fast uptake.
Emerging WHIM Syndrome Drugs
Plerixafor: National Institute of Allergy and Infectious Diseases (NIAID)
Plerixafor (MOZOBIL, AMD3100), approved by the US FDA, mobilizes CD34+ hematopoietic stem cells for autologous transplantation in non-Hodgkin's lymphoma and multiple myeloma patients. It also addresses WHIM syndrome by partially blocking CXCR4, used at lower doses than for stem cell mobilization. The National Institute of Allergy and Infectious Diseases is advancing plerixafor for WHIM syndrome, which has completed Phase III trials. Its mechanism involves specific, reversible inhibition of the CXCR4 receptor, disrupting the binding of stromal cell-derived factor-1 (SDF-1) and affecting cellular adhesion.
However, since the conclusion of Phase II/III trials in 2021, there have been no updates or further advancements reported for this drug. This stagnation suggests a potential pause or lack of progress in its development pipeline or regulatory status.
With first approved therapy for WHIM Syndrome in 2024, the market outlook for WHIM is expected to expand.
Treatment for WHIM syndrome includes both pharmacological and non-pharmacological approaches. Pharmacological treatments primarily involve Plerixafor (MOZOBIL), a CXCR4 receptor antagonist that helps manage neutropenia by partially blocking CXCR4 signaling. This is often used alongside Granulocyte-Colony Stimulating Factor (G-CSF) to enhance white blood cell mobilization. Additionally, antibiotics and antifungals are prescribed to manage infections stemming from the immune deficiencies characteristic of WHIM syndrome.
On the non-pharmacological side, supportive care is crucial, involving regular monitoring to manage and prevent infections. Lifestyle adjustments such as practicing good hygiene and minimizing infection risks are also recommended. Furthermore, psychosocial support is important to help patients cope with the emotional and psychological challenges associated with living with a chronic condition. These combined strategies aim to improve overall health outcomes and quality of life for individuals with WHIM syndrome.
WHIM Pipeline Development Activities
The report provides insights into WHIM syndrome clinical trials within Phase III, Phase II, and Phase I stage. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for WHIM emerging therapies.
KOL Views
To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate the secondary research. Industry Experts were contacted for insights on WHIM evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including KOL from Associate Research Physician, National Institute of Allergy and Infectious Disease, the US; University Hospital Aachen, Germany; Department of Pediatrics, Institute of Molecular Medicine, Brescia, Italy; Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan; Immunologist, Sant Joan de Deu Research, Spain, and others.
Delveinsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging therapies, treatment patterns, or WHIM market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Market Access and Reimbursement
The high cost of therapies for the treatment is a major factor restraining the growth of the global drug market. Because of the high cost, the economic burden is increasing, leading the patient to escape from proper treatment.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
Scope of the WHIM Syndrome Market Report
Key Questions Answered In The WHIM Syndrome Market Report
WHIM Syndrome Market Insights
WHIM Syndrome Epidemiology Insights
Current WHIM Syndrome Treatment Scenario, Marketed Drugs, and Emerging Therapies
Reasons to Buy WHIM Syndrome Market Report
The WHIM Epidemiology and Market Insight report for the 7MM covers the forecast period from 2024 to 2034, providing a projection of market dynamics and trends during this timeframe.
The WHIM market currently lacks robust development. However, the forecast period may see significant activity as major players are anticipated to enter the market and focus on developing treatments for WHIM syndrome.
The market size is estimated through data analysis, statistical modeling, and expert opinions. It may consider factors such as incident cases, treatment costs, revenue generated, and market trends.
The increase in diagnosed prevalent cases of WHIM and the launch of emerging therapies are attributed to be the key drivers for increasing the WHIM market.
Introducing new therapies, advancements in diagnostic techniques, and innovations in treatment approaches can significantly impact the WHIM treatment market. Market forecast reports may provide analysis and predictions regarding the potential impact of these developments.
The market forecast report may include information on the competitive landscape, profiling key market players, their product offerings, partnerships, and strategies, and helping stakeholders understand the competitive dynamics of the WHIM market.